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36 results

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Page 1
Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.
Hirawat S, Welch EM, Elfring GL, Northcutt VJ, Paushkin S, Hwang S, Leonard EM, Almstead NG, Ju W, Peltz SW, Miller LL. Hirawat S, et al. Among authors: almstead ng. J Clin Pharmacol. 2007 Apr;47(4):430-44. doi: 10.1177/0091270006297140. J Clin Pharmacol. 2007. PMID: 17389552 Clinical Trial.
PTC124 targets genetic disorders caused by nonsense mutations.
Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, Paushkin S, Patel M, Trotta CR, Hwang S, Wilde RG, Karp G, Takasugi J, Chen G, Jones S, Ren H, Moon YC, Corson D, Turpoff AA, Campbell JA, Conn MM, Khan A, Almstead NG, Hedrick J, Mollin A, Risher N, Weetall M, Yeh S, Branstrom AA, Colacino JM, Babiak J, Ju WD, Hirawat S, Northcutt VJ, Miller LL, Spatrick P, He F, Kawana M, Feng H, Jacobson A, Peltz SW, Sweeney HL. Welch EM, et al. Among authors: almstead ng. Nature. 2007 May 3;447(7140):87-91. doi: 10.1038/nature05756. Epub 2007 Apr 22. Nature. 2007. PMID: 17450125
Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.
Naryshkin NA, Weetall M, Dakka A, Narasimhan J, Zhao X, Feng Z, Ling KK, Karp GM, Qi H, Woll MG, Chen G, Zhang N, Gabbeta V, Vazirani P, Bhattacharyya A, Furia B, Risher N, Sheedy J, Kong R, Ma J, Turpoff A, Lee CS, Zhang X, Moon YC, Trifillis P, Welch EM, Colacino JM, Babiak J, Almstead NG, Peltz SW, Eng LA, Chen KS, Mull JL, Lynes MS, Rubin LL, Fontoura P, Santarelli L, Haehnke D, McCarthy KD, Schmucki R, Ebeling M, Sivaramakrishnan M, Ko CP, Paushkin SV, Ratni H, Gerlach I, Ghosh A, Metzger F. Naryshkin NA, et al. Among authors: almstead ng. Science. 2014 Aug 8;345(6197):688-93. doi: 10.1126/science.1250127. Science. 2014. PMID: 25104390
Ataluren Pharmacokinetics in Healthy Japanese and Caucasian Subjects.
Kong R, Laskin OL, Kaushik D, Jin F, Ma J, McIntosh J, Souza M, Almstead N. Kong R, et al. Clin Pharmacol Drug Dev. 2019 Feb;8(2):172-178. doi: 10.1002/cpdd.645. Epub 2019 Jan 10. Clin Pharmacol Drug Dev. 2019. PMID: 30629861 Free PMC article. Clinical Trial.
Targeting of Hematologic Malignancies with PTC299, A Novel Potent Inhibitor of Dihydroorotate Dehydrogenase with Favorable Pharmaceutical Properties.
Cao L, Weetall M, Trotta C, Cintron K, Ma J, Kim MJ, Furia B, Romfo C, Graci JD, Li W, Du J, Sheedy J, Hedrick J, Risher N, Yeh S, Qi H, Arasu T, Hwang S, Lennox W, Kong R, Petruska J, Moon YC, Babiak J, Davis TW, Jacobson A, Almstead NG, Branstrom A, Colacino JM, Peltz SW. Cao L, et al. Among authors: almstead ng. Mol Cancer Ther. 2019 Jan;18(1):3-16. doi: 10.1158/1535-7163.MCT-18-0863. Epub 2018 Oct 23. Mol Cancer Ther. 2019. PMID: 30352802 Free PMC article.
Identification of PTC725, an orally bioavailable small molecule that selectively targets the hepatitis C Virus NS4B protein.
Gu Z, Graci JD, Lahser FC, Breslin JJ, Jung SP, Crona JH, McMonagle P, Xia E, Liu S, Karp G, Zhu J, Huang S, Nomeir A, Weetall M, Almstead NG, Peltz SW, Tong X, Ralston R, Colacino JM. Gu Z, et al. Among authors: almstead ng. Antimicrob Agents Chemother. 2013 Jul;57(7):3250-61. doi: 10.1128/AAC.00527-13. Epub 2013 Apr 29. Antimicrob Agents Chemother. 2013. PMID: 23629699 Free PMC article.
36 results